Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…
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Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…
The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…
Hemophilia care providers in the U.K. have insufficient knowledge on how to assess the risk of prolonged bleeding associated with dental procedures, a study has found. The study, “U.K. hemophilia treaters’ knowledge of risk assessment for prolonged bleeding associated with dental procedures,” was published in the journal…
A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…
Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…
New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia to preventive treatment with FVIII, according to a small study. The study “Evaluation of EC50 of factor VIII as predictor of prophylaxis efficacy in patients…
Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, according to a real-world study. The study, “Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective…
People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved the prophylactic use of Hemlibra in patients of all ages who do not have factor VIII inhibitors, announced the treatment’s manufacturer, Roche. Hemlibra is a man-made antibody…
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact on…
